title: New and investigational treatments in cystic fibrosis.
abstract: Cystic fibrosis (CF) is an autosomal recessive disorder that affects approximately 1 in 3,000 Caucasian births, or 30,000 individuals in the US and 70,000 worldwide. The discovery of the CF gene, isolation of the CFTR protein and understanding of molecular mechanisms behind the clinical expression of CF are being translated into newer treatments. Treatments for CF and its manifestations are discussed in this article including inhaled antibiotics, hydrator therapies, anti-inflammatory agents and protein modifiers. New and experimental treatments that are in development are also discussed. Outcomes for these treatments are forced expiratory volume in one second (FEV(1)) improvement, CF-related quality of life, use of intravenous antibiotics and frequency of exacerbations and hospitalizations.
authors.affiliation: The Adult Cystic Fibrosis Center, Division of Pulmonary, Critical Care and Sleep Medicine North Shore University Hospital and Long Island Jewish Medical Center, New Hyde Park, NY 11040, USA. mnarasimhan@nshs.edu
authors.forename: Mangala
authors.initials: M
authors.lastname: Narasimhan
authors.forename: Rubin
authors.initials: R
authors.lastname: Cohen
mesh_terms: D000280:Administration, Inhalation; D000336:Aerosols; D000900:Anti-Bacterial Agents / Q000008:administration & dosage; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D003550:Cystic Fibrosis / Q000188:drug therapy*; D006801:Humans
chemical_list: D000336:Aerosols; D000900:Anti-Bacterial Agents; D000893:Anti-Inflammatory Agents